Orally active ceramide-specific glycosyltransferase and alpha-glucosidase I and II inhibitor.1 Rescues trafficking-deficient F508del-CFTR in human airway epithelial cells via inhibition of ER alpha-glucosidases I and II.2 Pharmacological chaperone for glucocerebrosidase degradation.3 Clinically useful agent for Gaucher disease type 1.4 Stabilizes neurological disorders in Niemann-Pick disease type C.5
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